A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

• ClinicalTrials.gov Identifier: NCT04360265
• Recruitment Status: Enrolling by invitation
• First Posted: April 24, 2020
• Last Update Posted: February 7, 2024
• Sponsor: Ultragenyx Pharmaceutical Inc
• Collaborator: Abeona Therapeutics, Inc
• Information provided by (Responsible Party): Ultragenyx Pharmaceutical Inc

Study Description

Brief Summary:

The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Condition or disease	Intervention/treatment	Phase
Mucopolysaccharidosis III-A	Other: No Investigational Product; Drug: Adjuvant Immunosuppression (IS) Therapy	Phase 3

Detailed Description:

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 33 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
• Actual Study Start Date: September 28, 2020
• Estimated Primary Completion Date: June 2027
• Estimated Study Completion Date: June 2027

Arms and Interventions

Arm	Intervention/treatment
Cohort 1; Participants who have completed a prior clinical trial involving the administration of ABO-102 and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IS therapy.	Other: No Investigational Product; No investigational product will be administered in this follow-up trial.; Drug: Adjuvant Immunosuppression (IS) Therapy; The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.
Cohort 2; Participants who have completed a prior clinical trial involving the administration of ABO-102 and who cannot participate in Cohort 1. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.	Other: No Investigational Product; No investigational product will be administered in this follow-up trial.

Outcome Measures

Primary Outcome Measures :

1. Adverse Events [ Time Frame: Up to Year 5 ]
   Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).

Secondary Outcome Measures :

1. Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS) [ Time Frame: Up to Month 12 ]
2. Change From Baseline (in Prior Trial) in the Cognitive Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
3. Change From Baseline (in Prior Trial) in the Language Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
4. Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II) [ Time Frame: Baseline, Up to Year 5 ]

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Participants that have completed a prior clinical trial involving the administration of ABO-102.
• Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Exclusion Criteria:

• Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during this study.
• Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Contacts and Locations

Locations

United States, Ohio

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Australia, South Australia

Women's and Children's Hospital

North Adelaide, South Australia, Australia

Spain

Hospital Clínico Universitario de Santiago

Santiago De Compostela, Spain

Sponsors and Collaborators

Ultragenyx Pharmaceutical Inc

Abeona Therapeutics, Inc

Investigators

• Study Director: Medical Director; Ultragenyx Pharmaceutical Inc

More Information

• Responsible Party: Ultragenyx Pharmaceutical Inc
• ClinicalTrials.gov Identifier: NCT04360265
• Other Study ID Numbers: LTFU-ABO-102; 2019-002979-34 ( EudraCT Number ); UX111-CL302 ( Other Identifier: Ultragenyx Pharmaceutical Inc )
• First Posted: April 24, 2020
• Last Update Posted: February 7, 2024
• Last Verified: February 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ultragenyx Pharmaceutical Inc:

MPS IIIA; Mucopolysaccharidosis IIIA; Sanfilippo A

Additional relevant MeSH terms:

Mucopolysaccharidoses; Mucopolysaccharidosis III; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Metabolic Diseases